Skin: New gene therapy cures serious diseases

Skin: New gene therapy cures serious diseases.


A gene therapy gel for blistering skin disease developed at Stanford Medicine worked wonders in a clinical trial.


The gel, called B-VEC, was intended to treat dystrophic epidermolysis bullosa, a skin disease that results in large, open sores that last for decades. The condition is extremely painful and medical treatment is mostly limited to palliative care. 


After four months, I saw improvement on a large wound on my back that I’d had for 20 years. After six months, the wound had healed completely and was much less painful.


Vincenzo Mascoli, 22 years old, participant in the study

This was an event that changed Vincenzo’s life. He now he can bathe and sleep on his back without pain. This treatment made a huge difference in the quality of life for Vincenzo and other trial participants.

Peter Marinkovich , MD, director of the Stanford Medicine Blistering Disease Clinic and senior author of the study

Patients with recessive dystrophic epidermolysis bullosa have a genetic mutation that prevents them from making a protein called collagen VII, which is known to bind the middle and outer layers of skin together. The absence of the protein causes the layers to flow through each other, resulting in blisters that form painful open sores.


The gel delivers a copy of the collagen VII gene to the skin’s surface. It does this by using a modified herpes simplex virus which, by making the protein missing, stabilizes the structure of the skin. The herpes virus has evolved to evade the human immune system, so the gel can be applied repeatedly without triggering an immune response.


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